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Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02468193
Recruitment Status : Completed
First Posted : June 10, 2015
Results First Posted : March 11, 2020
Last Update Posted : May 6, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Cushing's Syndrome
Ectopic Corticotropin Syndrome
Adrenal Adenoma
Adrenal Carcinoma
AIMAH
PPNAD
Intervention Drug: Osilodrostat
Enrollment 9
Recruitment Details Planned subjects: 10 subjects Actual enrolled: 9 subjects
Pre-assignment Details Planned subjects: 10 subjects
Arm/Group Title Osilodrostat
Hide Arm/Group Description Patients in this arm took the study drug, osilodrostat.
Period Title: Overall Study
Started 9
Completed Week-12 (Study Period I) 7
Discontinued at or Prior to Week-12 2
Completed Week-12, Did Not Enter Per. II 3
Completed Week-12, Entered Study Per. II 4
Completed Week-48 (Study Period II) 2
Discontinued at or Prior to Week48 2
Compl. Wk-48, Didn't Enter Opt. Ext. Per 0
Compl. Wk-48, Entered Opt. Ext. Period 2
Completed Optional Extension Period 0
Discontinued Study in Optional Ext. Per. 2
Completed 3
Not Completed 6
Reason Not Completed
Adverse Event             4
Subject/ Guardian decision             2
Arm/Group Title Osilodrostat
Hide Arm/Group Description Patients in this arm took the study drug, osilodrostat.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants
51.0  (18.17)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
7
  77.8%
Male
2
  22.2%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Japanese Number Analyzed 9 participants
9
Type of disease  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 9 participants
ACTH-Independent Macronodular Adrena;l Hyperplasia 1
Adrenal Adenoma 5
Ectopic Corticotropin Syndrome 3
1.Primary Outcome
Title Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12
Hide Description Percent change from baseline in the mUFC at the individual patient level
Time Frame Baseline, 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH- Patient 1 (n=1) Number Analyzed 1 participants
-99.0
Adrenal adenoma - Patient 1 (n=1) Number Analyzed 1 participants
-97.8
Adrenal adenoma - Patient 2 (n=1) Number Analyzed 1 participants
-94.5
Adrenal adenoma - Patient 3 (n=1) Number Analyzed 1 participants
-91.5
Adrenal adenoma - Patient 4 (n=1) Number Analyzed 1 participants
-81.8
Adrenal adenoma - Patient 5 (n=1) Number Analyzed 1 participants
-52.6
Ectopic corticotropin syndrome - Patient 1 (n=1) Number Analyzed 1 participants
-99.0
Ectopic c orticotropin syndrome - Patient 2(n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome - Patient 3 (n=0) Number Analyzed 0 participants
2.Secondary Outcome
Title Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)
Hide Description Percent change from baseline in the mUFC at the individual patient level
Time Frame Baseline, Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1: Week (Wk) 24 (n=1) Number Analyzed 1 participants
-99.5
AIMAH-Patient 1:Wk 48 (n=1) Number Analyzed 1 participants
-99.1
Adrenal adenoma-Patient 1: Wk 24(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 1: Wk 48(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 2: Wk 24(n=1) Number Analyzed 1 participants
-85.2
Adrenal adenoma-Patient 2: Wk 48(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 24(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 48(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 24(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 48(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 24(n=0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 48(n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 1: Wk 24(n=1) Number Analyzed 1 participants
-91.6
Ectopic corticotropin syndrome-Pt 1: Wk 48(n=1) Number Analyzed 1 participants
-91.0
Ectopic corticotropin syndrome-Pt 2: Wk 24(n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk 48(n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 24(n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 48(n=0) Number Analyzed 0 participants
3.Secondary Outcome
Title Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
Hide Description Absolute change from baseline in the mUFC
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: nmol/24hr
Week 12 (day 85)( n = 7) Number Analyzed 7 participants
-422.10
(-10487.5 to -157.0)
Week 24 (day 169) (n = 3) Number Analyzed 3 participants
-7428.10
(-9702.0 to -367.9)
Week 48 (day 337) (n = 2) Number Analyzed 2 participants
-8521.00
(-9640.5 to -7401.5)
4.Secondary Outcome
Title Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
Hide Description Percent change from baseline in the mUFC
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: percentage change
Week 12 (day 85)( n = 7) Number Analyzed 7 participants
-94.47
(-99.0 to -52.6)
Week 24 (day 169) (n = 3) Number Analyzed 3 participants
-91.57
(-99.5 to -85.2)
Week 48 (day 337) (n = 2) Number Analyzed 2 participants
-95.04
(-99.1 to -91.0)
5.Secondary Outcome
Title Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response
Hide Description Complete response rate = percentage of participants who had mUFC≤ ULN; Partial response rate = Percentage of participants who had mUFC>ULN and at least 50% reduction from baseline in mUFC. Overall response rate = Percentage of participants who had mUFC ≤ ULN or at least 50% reduction from baseline.
Time Frame 12, 24 and 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 9
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
Week 12: Complete responders Number Analyzed 9 participants
66.7
(29.9 to 92.5)
Week 12: Partial responders Number Analyzed 9 participants
11.1
(0.3 to 48.2)
Week 12: Overall responders Number Analyzed 9 participants
77.8
(40.0 to 97.2)
Week 24: Complete responders (n =3) Number Analyzed 3 participants
66.7
(9.4 to 99.2)
Week 24: Partial responders (n = 3) Number Analyzed 3 participants
33.3
(0.8 to 90.6)
Week 24: Overall responders (n = 3) Number Analyzed 3 participants
100
(29.2 to 100.0)
Week 48: Complete responders (n = 2) Number Analyzed 2 participants
50.0
(1.3 to 98.7)
Week 48: Partial responders (n = 2) Number Analyzed 2 participants
50.0
(1.3 to 98.7)
Week 48: Overall responders (n = 2) Number Analyzed 2 participants
100
(15.8 to 100.0)
6.Secondary Outcome
Title Absolute Change From Baseline in Morning Serum Cortisol at Individual Level
Hide Description Absolute change from baseline in morning serum cortisol at the individual patient level
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: nmol/L
AIMAH-Patient (Pt) 1: Week (Wk) 12 (n = 1) Number Analyzed 1 participants
-607
AIMAH-Patient 1: Wk 24 (n = 1) Number Analyzed 1 participants
-571
AIMAH-Patient 1: Wk 48 (n = 1) Number Analyzed 1 participants
-580
Adrenal adenoma-Patient 1: Wk 12 (n =1) Number Analyzed 1 participants
-334
Adrenal adenoma-Patient 1:Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 1: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 2: Wk 12(n =1) Number Analyzed 1 participants
-157
Adrenal adenoma-Patient 2: Wk 24 (n =1) Number Analyzed 1 participants
-135
Adrenal adenoma-Patient 2: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 12 (n =1) Number Analyzed 1 participants
-300
Adrenal adenoma-Patient 3: Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 12 (n =1) Number Analyzed 1 participants
-30
Adrenal adenoma-Patient 4: Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 12 (n =1) Number Analyzed 1 participants
-13
Adrenal adenoma-Patient 5: Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 48 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) Number Analyzed 1 participants
-949
Ectopic corticotropin syndrome-Pt 1: Wk24 (n=1) Number Analyzed 1 participants
-927
Ectopic corticotropin syndrome-Pt 1: Wk48 (n=1) Number Analyzed 1 participants
-861
Ectopic corticotropin syndrome-Pt 2: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk 24 (n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk48 (n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk24 (n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk48 (n=0) Number Analyzed 0 participants
7.Secondary Outcome
Title Percentage Change From Baseline in Morning Serum Cortisol at Individual Level
Hide Description Percentage change from baseline in morning serum cortisol at the individual patient level
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1: Week (Wk) 12 (n = 1) Number Analyzed 1 participants
-73.3
AIMAH-Patient 1: Wk 24 (n = 1) Number Analyzed 1 participants
-69.0
AIMAH-Patient 1: Wk 48 (n = 1) Number Analyzed 1 participants
-70.0
Adrenal adenoma-Patient 1: Wk 12 (n =1) Number Analyzed 1 participants
-78.0
Adrenal adenoma-Patient 1:Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 1: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 2: Wk 12(n =1) Number Analyzed 1 participants
-50.8
Adrenal adenoma-Patient 2: Wk 24 (n =1) Number Analyzed 1 participants
-43.7
Adrenal adenoma-Patient 2: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 12 (n =1) Number Analyzed 1 participants
-56.1
Adrenal adenoma-Patient 3: Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 12 (n =1) Number Analyzed 1 participants
-8.7
Adrenal adenoma-Patient 4: Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 12 (n =1) Number Analyzed 1 participants
-3.5
Adrenal adenoma-Patient 5: Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 48 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) Number Analyzed 1 participants
-71.4
Ectopic corticotropin syndrome-Pt 1: Wk24 (n=1) Number Analyzed 1 participants
-69.7
Ectopic corticotropin syndrome-Pt 1: Wk48 (n=1) Number Analyzed 1 participants
-64.7
Ectopic corticotropin syndrome-Pt 2: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk24 (n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk48 (n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk24 (n=0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk48 (n=0) Number Analyzed 0 participants
8.Secondary Outcome
Title Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level
Hide Description Absolute change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title ACTH Serum 11-deoxycorticosterone Aldosterone Estradiol
Hide Arm/Group Description:
Adrenocorticotropic hormone
adrenal steroid hormones: Serum 11-deoxycorticosterone
adrenal steroid hormones: Aldosterone
adrenal steroid hormones: Estradiol
Overall Number of Participants Analyzed 7 7 7 7
Measure Type: Number
Unit of Measure: pmol/L
AIMAH-Patient (Pt) 1 Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 2179 52 705
AIMAH-Pt 1 Wk 24 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 3601 236 235
AIMAH-Pt 1 Wk 48 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 454 170 400
Adrenal adenoma-Pt 1 Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
1.1 2966 -27 -404
Adrenal adenoma-Pt 1 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 5145 -981 4
Adrenal adenoma-Pt 2 Wk 24(n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 0 450 -4
Adrenal adenoma-Pt 2 Wk 48 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 0 -147 77
Adrenal adenoma-Pt 3 Wk 24 (n=0, 0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48(n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 787 3 18
Adrenal adenoma-Pt 4 Wk 24 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0 0 0 -11
Adrenal adenoma-Pt 5 Wk 24 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-67.7 -696 145 51
Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-47.3 -91 40 -11
Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
13.3 -91 37 11
Ectopic ACTH syndrome-Pt 2: Wk 12 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2: Wk 24 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2: Wk 48 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3: Wk 12 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3: Wk 24 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3: Wk 48 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
9.Secondary Outcome
Title Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level
Hide Description Percent change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title ACTH Serum 11-deoxycorticosterone Aldosterone Estradiol
Hide Arm/Group Description:
Adrenocorticotropic hormone
adrenal steroid hormones: Serum 11-deoxycorticosterone
adrenal steroid hormones: Aldosterone
adrenal steroid hormones: Estradiol
Overall Number of Participants Analyzed 7 7 7 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1 Wk 12 (n =0,1,0,1) Number Analyzed 0 participants 1 participants 0 participants 1 participants
118.0 1282
AIMAH-Pt 1 Wk 24 (n=0,1,0,1) Number Analyzed 0 participants 1 participants 0 participants 1 participants
195.1 427.3
AIMAH-Pt 1 Wk 48 (n=0,1,0,1) Number Analyzed 0 participants 1 participants 0 participants 1 participants
24.6 727.3
Adrenal adenoma-Pt 1 Wk 12 (n =0,0,1,1) Number Analyzed 0 participants 0 participants 1 participants 1 participants
-13.9 -53.7
Adrenal adenoma-Pt 1 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =0,0,1,1) Number Analyzed 0 participants 0 participants 1 participants 1 participants
-100 1.9
Adrenal adenoma-Pt 2 Wk 24 (n =0,0,1,1) Number Analyzed 0 participants 0 participants 1 participants 1 participants
45.9 -1.9
Adrenal adenoma-Pt 2 Wk 48 (n =0,0,0, 0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =0,0,1,1) Number Analyzed 0 participants 0 participants 1 participants 1 participants
-75.0 513.3
Adrenal adenoma-Pt 3 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =0,0,1,1) Number Analyzed 0 participants 0 participants 1 participants 1 participants
10.0 120.0
Adrenal adenoma-Pt 4 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =0,0,0,1) Number Analyzed 0 participants 0 participants 0 participants 1 participants
-100
Adrenal adenoma-Pt 5 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,0,1) Number Analyzed 1 participants 1 participants 0 participants 1 participants
-71.6 -100 72.9
Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,0,1) Number Analyzed 1 participants 1 participants 0 participants 1 participants
-50.0 -13.1 -15.7
Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,0,1) Number Analyzed 1 participants 1 participants 0 participants 1 participants
14.1 -13.1 15.7
Ectopic ACTH syndrome-Pt 2: Wk 12 (n=0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
10.Secondary Outcome
Title Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels
Hide Description Absolute change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Serum 11-deoxycortisol Testosterone
Hide Arm/Group Description:
adrenal steroid hormones: Serum 11-deoxycortisol
adrenal steroid hormones: Testosterone
Overall Number of Participants Analyzed 7 7
Measure Type: Number
Unit of Measure: nmol/L
AIMAH-Patient (Pt) 1 Wk 12 (n = 1,1) Number Analyzed 1 participants 1 participants
-4.35 0.76
AIMAH-Pt 1 Wk 24 (n = 1,1) Number Analyzed 1 participants 1 participants
-8.5 0.66
AIMAH-Pt 1 Wk 48 (n = 1,1) Number Analyzed 1 participants 1 participants
-15.54 -0.03
Adrenal adenoma-Pt 1 Wk 12 (n = 1,1) Number Analyzed 1 participants 1 participants
6.91 0.31
Adrenal adenoma-Pt 1 Wk 24 (n =0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n = 1,1) Number Analyzed 1 participants 1 participants
2.81 0.11
Adrenal adenoma-Pt 2 Wk 24 (n = 1,1) Number Analyzed 1 participants 1 participants
-0.64 -0.35
Adrenal adenoma-Pt 2 Wk 48 (n = 0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n = 1,1) Number Analyzed 1 participants 1 participants
7.34 0.42
Adrenal adenoma-Pt 3 Wk 24 (n = 0, 0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n = 1,1) Number Analyzed 1 participants 1 participants
3.5 0.03
Adrenal adenoma-Pt 4 Wk 24 (n = 0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n = 0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n = 1,1) Number Analyzed 1 participants 1 participants
-1.39 0
Adrenal adenoma-Pt 5 Wk 24 (n =0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n = 0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n = 1,1) Number Analyzed 1 participants 1 participants
-15.81 5.83
Ectopic ACTH syndrome-Pt 1 Wk 24 (n = 1,1) Number Analyzed 1 participants 1 participants
-12.41 -2.71
Ectopic ACTH syndrome-Pt 1 Wk 48 (n = 1,1) Number Analyzed 1 participants 1 participants
-2.52 -2.01
Ectopic ACTH syndrome-Pt 2 Wk 12 (n = 0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n = 0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n = 0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12(n =0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n = 0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48(n = 0,0) Number Analyzed 0 participants 0 participants
11.Secondary Outcome
Title Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels
Hide Description Percent change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Serum 11-deoxycortisol Testosterone
Hide Arm/Group Description:
adrenal steroid hormones: Serum 11-deoxycortisol
adrenal steroid hormones: Testosterone
Overall Number of Participants Analyzed 7 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1 Wk 12 (n=1, 1) Number Analyzed 1 participants 1 participants
-17.8 73.1
AIMAH-Pt 1 Wk 24 (n=1, 1) Number Analyzed 1 participants 1 participants
-34.9 63.5
AIMAH-Pt 1 Wk 48 (n=1, 1) Number Analyzed 1 participants 1 participants
-63.7 -2.9
Adrenal adenoma-Pt 1 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
143.7 100.0
Adrenal adenoma-Pt 1 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
108.9 10.6
Adrenal adenoma-Pt 2 Wk 24 (n=1,1) Number Analyzed 1 participants 1 participants
-24.8 -33.7
Adrenal adenoma-Pt 2 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1, 1) Number Analyzed 1 participants 1 participants
127.2 93.3
Adrenal adenoma-Pt 3 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0,0 Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1, 1) Number Analyzed 1 participants 1 participants
267.2 14.3
Adrenal adenoma-Pt 4 Wk 24 (n=0,0 Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0,0 Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-43.2 0.0
Adrenal adenoma-Pt 5 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1, 1) Number Analyzed 1 participants 1 participants
-64.8 96.5
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1, 1) Number Analyzed 1 participants 1 participants
-50.9 -44.9
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1, 1) Number Analyzed 1 participants 1 participants
-10.3 -33.3
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12(n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
12.Secondary Outcome
Title Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level
Hide Description Absolute change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: mmol/L
AIMAH-Patient (Pt) 1 Wk 12 (n =1) Number Analyzed 1 participants
-0.78
AIMAH-Patient (Pt) 1 Wk 24 (n =1) Number Analyzed 1 participants
-0.84
AIMAH-Patient (Pt) 1 Wk 48 (n =1) Number Analyzed 1 participants
-0.78
Adrenal adenoma-Pt 1 Wk 12 (n =1) Number Analyzed 1 participants
1.22
Adrenal adenoma-Pt 1 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =1) Number Analyzed 1 participants
-1.89
Adrenal adenoma-Pt 2 Wk 24 (n =1) Number Analyzed 1 participants
0.22
Adrenal adenoma-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =1) Number Analyzed 1 participants
-0.28
Adrenal adenoma-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =1) Number Analyzed 1 participants
-0.67
Adrenal adenoma-Pt 4 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =1) Number Analyzed 1 participants
-0.05
Adrenal adenoma-Pt 5 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n =1) Number Analyzed 1 participants
-0.33
Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) Number Analyzed 1 participants
-0.39
Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) Number Analyzed 1 participants
0.06
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
13.Secondary Outcome
Title Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level
Hide Description Percentasge change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1 Wk 12 (n =1) Number Analyzed 1 participants
-15.3
AIMAH-Patient (Pt) 1 Wk 24 (n =1) Number Analyzed 1 participants
-16.4
AIMAH-Patient (Pt) 1 Wk 48 (n =1) Number Analyzed 1 participants
-15.3
Adrenal adenoma-Pt 1 Wk 12 (n =1) Number Analyzed 1 participants
24.2
Adrenal adenoma-Pt 1 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =1) Number Analyzed 1 participants
-27.2
Adrenal adenoma-Pt 2 Wk 24 (n =1) Number Analyzed 1 participants
3.2
Adrenal adenoma-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =1) Number Analyzed 1 participants
-5.0
Adrenal adenoma-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =1) Number Analyzed 1 participants
-11.3
Adrenal adenoma-Pt 4 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =1) Number Analyzed 1 participants
-0.9
Adrenal adenoma-Pt 5 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n =1) Number Analyzed 1 participants
-5.9
Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) Number Analyzed 1 participants
-7.0
Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) Number Analyzed 1 participants
1.1
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
14.Secondary Outcome
Title Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level
Hide Description Absolute change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage of HbA1c
AIMAH-Patient (Pt) 1 Wk 12 (n=1) Number Analyzed 1 participants
-0.1
AIMAH-Pt 1 Wk 24 (n=1) Number Analyzed 1 participants
-0.1
AIMAH-Pt 1 Wk 48 (n=1) Number Analyzed 1 participants
-0.2
Adrenal adenoma-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
0.3
Adrenal adenoma-Pt 1 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1) Number Analyzed 1 participants
-0.4
Adrenal adenoma-Pt 2 Wk 24 (n=1) Number Analyzed 1 participants
-0.7
Adrenal adenoma-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1) Number Analyzed 1 participants
-1.4
Adrenal adenoma-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1) Number Analyzed 1 participants
-0.9
Adrenal adenoma-Pt 4 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1) Number Analyzed 1 participants
-0.1
Adrenal adenoma-Pt 5 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
0.3
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) Number Analyzed 1 participants
0.5
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) Number Analyzed 1 participants
0.8
Ectopic ACTH syndrome-Pt 2 Wk 12(n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12(n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
15.Secondary Outcome
Title Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level
Hide Description Percentage change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1 Wk 12 (n=1) Number Analyzed 1 participants
-1.8
AIMAH-Pt 1 Wk 24 (n=1) Number Analyzed 1 participants
-1.8
AIMAH-Pt 1 Wk 48 (n=1) Number Analyzed 1 participants
-3.6
Adrenal adenoma-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
6.2
Adrenal adenoma-Pt 1 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1) Number Analyzed 1 participants
-6.5
Adrenal adenoma-Pt 2 Wk 24 (n=1) Number Analyzed 1 participants
-11.3
Adrenal adenoma-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1) Number Analyzed 1 participants
-20.3
Adrenal adenoma-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1) Number Analyzed 1 participants
-12.2
Adrenal adenoma-Pt 4 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1) Number Analyzed 1 participants
-1.5
Adrenal adenoma-Pt 5 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
5.4
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) Number Analyzed 1 participants
8.9
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) Number Analyzed 1 participants
14.3
Ectopic ACTH syndrome-Pt 2 Wk 12(n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12(n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
16.Secondary Outcome
Title Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level
Hide Description Absolute change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol & triglycerides, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Cholesterol HDL Cholesterol LDL Cholesterol Triglycerides
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7 7 7 7
Measure Type: Number
Unit of Measure: mmol/L
AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.67 -0.85 0.21 -0.87
AIMAH-Pt 1 Wk 24 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.65 -1.01 0.18 -0.64
AIMAH-Pt 1 Wk 48 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.72 -0.57 -0.08 -0.76
Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-1.56 -0.26 -1.11 -1.42
Adrenal adenoma-Pt 1 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.54 -0.64 -0.26 0.11
Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
0.26 -0.33 0.31 0.08
Adrenal adenoma-Pt 2 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.55 -0.16 -0.19 -1.36
Adrenal adenoma-Pt 3 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
1.09 0.18 0.72 0.58
Adrenal adenoma-Pt 4 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.39 -0.21 -0.18 0.44
Adrenal adenoma-Pt 5 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.16 0.16 -0.11 -0.47
Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.26 0.49 -0.29 -0.83
Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-0.08 0.29 -0.31 0.22
Ectopic ACTH syndrome-Pt 2 Wk 12 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0,0,0,0,) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
17.Secondary Outcome
Title Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level
Hide Description Percent change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol & triglycerides, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Cholesterol HDL Cholesterol LDL Cholesterol Triglycerides
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7 7 7 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-11.1 -33.2 6.3 -49.4
AIMAH-Pt 1 Wk 24 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-10.7 -39.5 5.4 -36.4
AIMAH-Pt 1 Wk 48 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-11.9 -22.3 -2.4 -43.2
Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-23.0 -27.1 -24.9 -28.8
Adrenal adenoma-Pt 1 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-11.9 -34.4 -10.6 12.5
Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
5.7 -17.7 12.6 9.1
Adrenal adenoma-Pt 2 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-9.5 -9.4 -5.5 -56.7
Adrenal adenoma-Pt 3 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
23.0 15.5 26.5 46.8
Adrenal adenoma-Pt 4 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-8.7 -11.4 -9.3 33.3
Adrenal adenoma-Pt 5 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-3.8 12.4 -4.5 -32.0
Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-6.2 38.0 -11.8 -56.5
Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) Number Analyzed 1 participants 1 participants 1 participants 1 participants
-1.9 22.5 -12.6 15.0
Ectopic ACTH syndrome-Pt 2 Wk 12 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0,0,0,0) Number Analyzed 0 participants 0 participants 0 participants 0 participants
18.Secondary Outcome
Title Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level
Hide Description Absolute change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: kg/m^2
AIMAH-Patient (Pt) 1 Wk 12 (n =1) Number Analyzed 1 participants
-0.5
AIMAH-Pt 1 Wk 24 (n =1) Number Analyzed 1 participants
-2.3
AIMAH-Pt 1 Wk 48 (n =1) Number Analyzed 1 participants
-1.6
Adrenal adenoma-Pt 1 Wk 12 (n =1) Number Analyzed 1 participants
0.6
Adrenal adenoma-Pt 1 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =1) Number Analyzed 1 participants
-1.2
Adrenal adenoma-Pt 2 Wk 24 (n =1) Number Analyzed 1 participants
-3.6
Adrenal adenoma-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =1) Number Analyzed 1 participants
0.7
Adrenal adenoma-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =1) Number Analyzed 1 participants
0.2
Adrenal adenoma-Pt 4 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =1) Number Analyzed 1 participants
0.1
Adrenal adenoma-Pt 5 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n =1) Number Analyzed 1 participants
1.8
Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) Number Analyzed 1 participants
3.0
Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) Number Analyzed 1 participants
3.6
Ectopic ACTH syndrome-Pt 2 Wk 12(n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
19.Secondary Outcome
Title Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level
Hide Description Percent change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1 Wk 12 (n =1) Number Analyzed 1 participants
-2.1
AIMAH-Pt 1 Wk 24 (n =1) Number Analyzed 1 participants
-9.6
AIMAH-Pt 1 Wk 48 (n =1) Number Analyzed 1 participants
-6.7
Adrenal adenoma-Pt 1 Wk 12 (n =1) Number Analyzed 1 participants
1.9
Adrenal adenoma-Pt 1 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n =1) Number Analyzed 1 participants
-3.1
Adrenal adenoma-Pt 2 Wk 24 (n =1) Number Analyzed 1 participants
-9.4
Adrenal adenoma-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n =1) Number Analyzed 1 participants
3.3
Adrenal adenoma-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n =1) Number Analyzed 1 participants
0.9
Adrenal adenoma-Pt 4 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n =1) Number Analyzed 1 participants
0.5
Adrenal adenoma-Pt 5 Wk 24 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n =1) Number Analyzed 1 participants
7.4
Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) Number Analyzed 1 participants
12.3
Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) Number Analyzed 1 participants
14.8
Ectopic ACTH syndrome-Pt 2 Wk 12(n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n =0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n =0) Number Analyzed 0 participants
20.Secondary Outcome
Title Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level
Hide Description Absolute change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: cm
AIMAH-Patient (Pt)1 Wk 12 (n=1) Number Analyzed 1 participants
-2.0
AIMAH-Pt1 Wk 24 (n=1) Number Analyzed 1 participants
-7.5
AIMAH-Pt1 Wk 48 (n=1) Number Analyzed 1 participants
-5.5
Adrenal adenoma-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
-2.0
Adrenal adenoma-Pt 1 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1) Number Analyzed 1 participants
-1.0
Adrenal adenoma-Pt 2 Wk 24 (n=1) Number Analyzed 1 participants
-8.0
Adrenal adenoma-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1) Number Analyzed 1 participants
-4.0
Adrenal adenoma-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1) Number Analyzed 1 participants
1.0
Adrenal adenoma-Pt 4 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1) Number Analyzed 1 participants
-0.3
Adrenal adenoma-Pt 5 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
2.5
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) Number Analyzed 1 participants
11.0
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) Number Analyzed 1 participants
9.5
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12(n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
21.Secondary Outcome
Title Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level
Hide Description Percent change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt)1 Wk 12 (n=1) Number Analyzed 1 participants
-2.4
AIMAH-Pt1 Wk 24 (n=1) Number Analyzed 1 participants
-9.0
AIMAH-Pt1 Wk 48 (n=1) Number Analyzed 1 participants
-6.6
Adrenal adenoma-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
-1.8
Adrenal adenoma-Pt 1 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1) Number Analyzed 1 participants
-0.9
Adrenal adenoma-Pt 2 Wk 24 (n=1) Number Analyzed 1 participants
-7.1
Adrenal adenoma-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1) Number Analyzed 1 participants
-4.9
Adrenal adenoma-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1) Number Analyzed 1 participants
1.1
Adrenal adenoma-Pt 4 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1) Number Analyzed 1 participants
-0.4
Adrenal adenoma-Pt 5 Wk 24 (n=0) Number Analyzed 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) Number Analyzed 1 participants
2.9
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) Number Analyzed 1 participants
12.9
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) Number Analyzed 1 participants
11.1
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12(n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0) Number Analyzed 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0) Number Analyzed 0 participants
22.Secondary Outcome
Title Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level
Hide Description Absolute change in cardiovascular-related metabolic parameter: sitting systolic BP & sitting diastolic BP, associated with Cushing's syndrome (CS)
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Sitting Systolic BP Sitting Diastolic BP
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7 7
Measure Type: Number
Unit of Measure: mmHg
AIMAH-Patient (Pt) 1Wk 12 (n =1, 1) Number Analyzed 1 participants 1 participants
-44.3 -31.0
AIMAH-Patient (Pt) 1 Wk 24 (n =1, 1) Number Analyzed 1 participants 1 participants
-48.34 -32.66
AIMAH-Patient (Pt) 1 Wk 48 (n =1, 1) Number Analyzed 1 participants 1 participants
-40.34 -19.0
Adrenal adenoma-Pt 1 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
11.67 -3.67
Adrenal adenoma-Pt 1 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-50.67 -37.0
Adrenal adenoma-Pt 2 Wk 24 (n=1,1) Number Analyzed 1 participants 1 participants
-32.33 -26.66
Adrenal adenoma-Pt 2 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-16.0 -6.67
Adrenal adenoma-Pt 3 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-6.67 7.33
Adrenal adenoma-Pt 4 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
11.0 3.33
Adrenal adenoma-Pt 5 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n=1,1) Number Analyzed 1 participants 1 participants
-17.67 -13.67
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1,1) Number Analyzed 1 participants 1 participants
4.33 4.0
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1,1) Number Analyzed 1 participants 1 participants
-1.0 -0.67
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
23.Secondary Outcome
Title Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level
Hide Description Percentage change in cardiovascular-related metabolic parameter: sitting systolic BP & sitting diastolic BP, associated with Cushing's syndrome
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Sitting Systolic BP Sitting Diastolic BP
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7 7
Measure Type: Number
Unit of Measure: percentage change
AIMAH-Patient (Pt) 1Wk 12 (n =1, 1) Number Analyzed 1 participants 1 participants
-31.1 -32.5
AIMAH-Patient (Pt) 1 Wk 24 (n =1, 1) Number Analyzed 1 participants 1 participants
-33.9 -34.3
AIMAH-Patient (Pt) 1 Wk 48 (n =1, 1) Number Analyzed 1 participants 1 participants
-28.3 -19.9
Adrenal adenoma-Pt 1 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
9.2 -4.1
Adrenal adenoma-Pt 1 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 1 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 2 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-31.3 -33.2
Adrenal adenoma-Pt 2 Wk 24 (n=1,1) Number Analyzed 1 participants 1 participants
-20.0 -23.9
Adrenal adenoma-Pt 2 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-12.3 -8.9
Adrenal adenoma-Pt 3 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 3 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
-4.3 9.9
Adrenal adenoma-Pt 4 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 4 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 12 (n=1,1) Number Analyzed 1 participants 1 participants
9.3 5.1
Adrenal adenoma-Pt 5 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Adrenal adenoma-Pt 5 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 1 Wk 12(n=1,1) Number Analyzed 1 participants 1 participants
-14.1 -16.5
Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1,1) Number Analyzed 1 participants 1 participants
3.4 4.8
Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1,1) Number Analyzed 1 participants 1 participants
-0.8 -0.8
Ectopic ACTH syndrome-Pt 2 Wk 12 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 2 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 12 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 24 (n=0,0) Number Analyzed 0 participants 0 participants
Ectopic ACTH syndrome-Pt 3 Wk 48 (n=0,0) Number Analyzed 0 participants 0 participants
24.Secondary Outcome
Title Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level
Hide Description The Cushing's Disease Health-Related Quality of Life Questionnaire (Cushing QoL) (version 1.0) was developed to evaluate quality of life in patients with Cushing's syndrome (Webb et al 2008). The Cushing QoL is comprised of 12 items that capture patient responses on seven concepts: daily activities, healing and pain, mood and self-confidence, social concerns, physical appearance, memory and concern about the future. Each questionnaire of the Cushing QOL has a scale of 1-5 where '1' corresponding to 'Always' or 'Very much' and '5' to 'Never' or 'Not at all'. The lower the score, the greater the impact on HRQoL. The score is the sum of all the item response and can range from 12 (worst) to 60 points (best).
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: scores on a scale
AIMAH-Patient 1: Week (Wk) 12 (n=1) Number Analyzed 1 participants
48
AIMAH-Patient 1: Wk 24 (n=1) Number Analyzed 1 participants
51
AIMAH-Patient 1: Wk 48 (n=1) Number Analyzed 1 participants
47
Adrenal adenoma-Patient 1: Wk 12(n=1) Number Analyzed 1 participants
29
Adrenal adenoma-Patient 1: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 1: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 2: Wk 12(n=1) Number Analyzed 1 participants
18
Adrenal adenoma-Patient 2: Wk 24 (n = 1) Number Analyzed 1 participants
22
Adrenal adenoma-Patient 2: Wk 48 (n =0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 12 (n =1) Number Analyzed 1 participants
29
Adrenal adenoma-Patient 3: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 12 (n =1) Number Analyzed 1 participants
38
Adrenal adenoma-Patient 4: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 12 (n=1) Number Analyzed 1 participants
45
Adrenal adenoma-Patient 5: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 48 (n = 0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) Number Analyzed 1 participants
31
Ectopic corticotropin syndrome-Pt 1: Wk 24 (n =1) Number Analyzed 1 participants
33
Ectopic corticotropin syndrome-Pt 1: Wk 48 (n =1) Number Analyzed 1 participants
23
Ectopic corticotropin syndrome-Pt 2: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk 24 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk 48 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 12 n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 24 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 48 (n =0) Number Analyzed 0 participants
25.Secondary Outcome
Title Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Beck Depression Inventory II (BDI-ll) Depression Score at Individual Level
Hide Description The Beck Depression Inventory II (BDI-II) is a patient reported instrument that consists of 21 items designed to assess the intensity of depression in clinical & normal patients in the preceding two weeks. Each item is a list of four statements arranged in increasing severity about a particular symptom of depression. Each of 21 items corresponds to a symptom of depression and the sum of total score will be calculated where each item has a four-point scale ranging from 0 to 3, leading to a total score from zero to 63.
Time Frame Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
Arm/Group Title Osilodrostat
Hide Arm/Group Description:
Patients in this arm took the study drug, osilodrostat.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: scores on a scale
AIMAH-Patient 1: Week (Wk) 12 (n =1) Number Analyzed 1 participants
4
AIMAH-Patient 1: Wk 24 (n =1) Number Analyzed 1 participants
15
AIMAH-Patient 1: Wk 48 (n =1) Number Analyzed 1 participants
12
Adrenal adenoma-Patient 1: Wk 12 (n =1) Number Analyzed 1 participants
33
Adrenal adenoma-Patient 1: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 1: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 2: Wk 12 (n =1) Number Analyzed 1 participants
35
Adrenal adenoma-Patient 2: Wk 24 (n =1) Number Analyzed 1 participants
37
Adrenal adenoma-Patient 2: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 12 (n =1) Number Analyzed 1 participants
30
Adrenal adenoma-Patient 3: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 3: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 12 (n =1) Number Analyzed 1 participants
26
Adrenal adenoma-Patient 4: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 4: Wk 48 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 12 (n =1) Number Analyzed 1 participants
10
Adrenal adenoma-Patient 5: Wk 24 (n = 0) Number Analyzed 0 participants
Adrenal adenoma-Patient 5: Wk 48 (n = 0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) Number Analyzed 1 participants
9
Ectopic corticotropin syndrome-Pt 1: Wk 24 (n =1) Number Analyzed 1 participants
10
Ectopic corticotropin syndrome-Pt 1: Wk 48 (n =1) Number Analyzed 1 participants
22
Ectopic corticotropin syndrome-Pt 2: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk 24 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 2: Wk 48 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 12 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 24 (n =0) Number Analyzed 0 participants
Ectopic corticotropin syndrome-Pt 3: Wk 48 (n =0) Number Analyzed 0 participants
26.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 0
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 0
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 1 8 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 0: 0.75 hour post-dose 0.971 [1]   (NA) 0.405  (0.673)
Week 0: 2 hours post-dose 5.11 [1]   (NA) 5.29  (4.94)
Week 0: 4 hours post-dose 3.77 [1]   (NA) 8.12  (2.36)
[1]
N/A : not enough participants to calculate the standard deviation
27.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 1
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 1, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 1 5 1 2
Mean (Standard Deviation)
Unit of Measure: ng/mL
10.9 [1]   (NA) 10.5  (6.57) 21.3 [1]   (NA) 22.3  (14.2)
[1]
N/A : not enough participants to calculate the standard deviation
28.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 2
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 2 2 2 2
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 2: 0.75 hour post-dose Number Analyzed 2 participants 2 participants 2 participants 2 participants
3.89  (2.76) 3.69  (3.3) 33.1  (19.2) 34.9  (34.6)
Week 2: 2 hours post-dose Number Analyzed 2 participants 2 participants 2 participants 2 participants
8.93  (2.36) 14.3  (7.64) 21.3  (4.53) 33.6  (8.84)
Week 2: 4 hours post-dose Number Analyzed 2 participants 1 participants 2 participants 2 participants
7.01  (2.69) 11.1 [1]   (NA) 13.6  (3.89) 29.2  (0.354)
[1]
N/A : not enough participants to calculate the standard deviation
29.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 3
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 3, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 4 1 1 2
Mean (Standard Deviation)
Unit of Measure: ng/mL
7.09  (2.07) 7.62 [1]   (NA) 23.2 [1]   (NA) 37.8  (0.849)
[1]
N/A : not enough participants to calculate the standard deviation
30.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 4
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 4, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 4 2 0 2
Mean (Standard Deviation)
Unit of Measure: ng/mL
4.08  (2.61) 9.91  (3.53) 24.0  (21.00)
31.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 6
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 6, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 4 2 0 1
Mean (Standard Deviation)
Unit of Measure: ng/mL
6.74  (2.42) 12.7  (9.08) 31.7 [1]   (NA)
[1]
N/A : not enough participants to calculate the standard deviation
32.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 8
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 8, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 5 1 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
3.35  (2.75) 13.9 [1]   (NA)
[1]
N/A : not enough participants to calculate the standard deviation
33.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 10
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 10, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 4 2 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
4.73  (1.26) 11.3  (4.57)
34.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 12
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 2 1 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 12: 0.75 hour post-dose 0.382  (0.171) 3.1 [1]   (NA)
Week 12: 2 hours post-dose 4.62  (2.51) 9.98 [1]   (NA)
Week 12: 4 hours post-dose 3.58  (2.3) 6.57 [1]   (NA)
[1]
N/A : not enough participants to calculate the standard deviation
35.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 16
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 16, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 2 0 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
5.71  (1.33)
36.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 20
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 20, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 1 0 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
0.794 [1]   (NA)
[1]
N/A : not enough participants to calculate the standard deviation
37.Secondary Outcome
Title Plasma Concentrations of Osilodrostat (LCI699) at Week 24
Hide Description Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Time Frame Week 24, 2 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
Arm/Group Title Osilodrostat 1mg Osilodrostat 2mg Osilodrostat 3mg Osilodrostat 5mg
Hide Arm/Group Description:
Patients in this arm took 1mg of study drug, osilodrostat.
Patients in this arm took 2mg of study drug, osilodrostat.
Patients in this arm took 3mg of study drug, osilodrostat.
Patients in this arm took 5mg of study drug, osilodrostat.
Overall Number of Participants Analyzed 2 0 0 0
Mean (Standard Deviation)
Unit of Measure: ng/mL
6.51  (2.14)
Time Frame Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks.
Adverse Event Reporting Description AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
 
Arm/Group Title Osilodrostat
Hide Arm/Group Description Patients in this arm took the study drug, osilodrostat.
All-Cause Mortality
Osilodrostat
Affected / at Risk (%)
Total   0/9 (0.00%) 
Hide Serious Adverse Events
Osilodrostat
Affected / at Risk (%)
Total   4/9 (44.44%) 
Cardiac disorders   
Myocardial infarction  1  1/9 (11.11%) 
Endocrine disorders   
Adrenal insufficiency  1  2/9 (22.22%) 
Infections and infestations   
Pneumonia  1  1/9 (11.11%) 
Psychiatric disorders   
Psychiatric symptom  1  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (21.1)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Osilodrostat
Affected / at Risk (%)
Total   9/9 (100.00%) 
Blood and lymphatic system disorders   
Iron deficiency anaemia  1  1/9 (11.11%) 
Ear and labyrinth disorders   
Vertigo  1  1/9 (11.11%) 
Endocrine disorders   
Adrenal insufficiency  1  5/9 (55.56%) 
Steroid withdrawal syndrome  1  1/9 (11.11%) 
Gastrointestinal disorders   
Abdominal distension  1  1/9 (11.11%) 
Abdominal pain upper  1  1/9 (11.11%) 
Constipation  1  2/9 (22.22%) 
Dental caries  1  1/9 (11.11%) 
Enterocolitis  1  1/9 (11.11%) 
Stomatitis  1  1/9 (11.11%) 
General disorders   
Malaise  1  3/9 (33.33%) 
Oedema peripheral  1  1/9 (11.11%) 
Pyrexia  1  1/9 (11.11%) 
Hepatobiliary disorders   
Primary biliary cholangitis  1  1/9 (11.11%) 
Infections and infestations   
Cellulitis  1  1/9 (11.11%) 
Nasopharyngitis  1  3/9 (33.33%) 
Osteomyelitis  1  1/9 (11.11%) 
Injury, poisoning and procedural complications   
Rib fracture  1  1/9 (11.11%) 
Investigations   
Alanine aminotransferase increased  1  2/9 (22.22%) 
Amylase increased  1  1/9 (11.11%) 
Aspartate aminotransferase increased  1  2/9 (22.22%) 
Blood alkaline phosphatase increased  1  2/9 (22.22%) 
Gamma-glutamyltransferase increased  1  3/9 (33.33%) 
Weight decreased  1  1/9 (11.11%) 
Weight increased  1  1/9 (11.11%) 
Metabolism and nutrition disorders   
Decreased appetite  1  1/9 (11.11%) 
Dyslipidaemia  1  1/9 (11.11%) 
Hypokalaemia  1  2/9 (22.22%) 
Hypomagnesaemia  1  1/9 (11.11%) 
Increased appetite  1  1/9 (11.11%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/9 (11.11%) 
Nervous system disorders   
Dizziness  1  1/9 (11.11%) 
Psychiatric disorders   
Anxiety  1  1/9 (11.11%) 
Insomnia  1  1/9 (11.11%) 
Mood altered  1  1/9 (11.11%) 
Nightmare  1  1/9 (11.11%) 
Reactive psychosis  1  1/9 (11.11%) 
Skin and subcutaneous tissue disorders   
Alopecia  1  1/9 (11.11%) 
Dermal cyst  1  1/9 (11.11%) 
Dermatitis acneiform  1  2/9 (22.22%) 
Pruritus  1  2/9 (22.22%) 
Rash  1  2/9 (22.22%) 
Vascular disorders   
Peripheral coldness  1  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (21.1)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis PharmaCeuticals
Phone: 862-778-8300
EMail: novartis.email@novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02468193    
Other Study ID Numbers: CLCI699C1201
First Submitted: June 8, 2015
First Posted: June 10, 2015
Results First Submitted: October 25, 2019
Results First Posted: March 11, 2020
Last Update Posted: May 6, 2020